On May 24, 2021 Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, reported that Boehringer Ingelheim has accepted a GalXC RNAi candidate for advancement under the existing agreement between the companies for the discovery and development of novel therapies for the treatment of chronic liver diseases (Press release, Boehringer Ingelheim, MAY 24, 2021, View Source [SID1234580496]). Referred to as DCR-LIV2, the compound will be investigated for the treatment of nonalcoholic steatohepatitis (NASH), a chronic liver disease for which there are no approved therapeutic interventions. Acceptance of DCR-LIV2 as a development candidate triggered a single-digit multimillion USD preclinical milestone payment to Dicerna, which the Company expects to receive in the second quarter of 2021
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Advancing this selected target to development demonstrates the commitment of our team and our collaborative partners at Boehringer Ingelheim to discovering new and innovative ways to treat NASH using RNAi," said Bob D. Brown, Ph.D., Dicerna’s Chief Scientific Officer and Executive Vice President of R&D. "Dicerna’s GalXC technology is the ideal platform for this vital work. What’s more, this announcement marks an important milestone for Dicerna and highlights the productivity of our RNAi discovery research engine, as all of Dicerna’s discovery-oriented collaborations have now produced GalXC-based development candidates that are advancing toward the clinic or have already entered clinical development."
Under the terms of the existing agreement between Dicerna and Boehringer Ingelheim, Dicerna is eligible to receive up to $170.0 million in potential additional development and commercial milestones related to DCR-LIV2. Dicerna is also eligible to receive tiered mid-single-digit royalties on potential global net sales.
About Nonalcoholic Steatohepatitis (NASH)
Nonalcoholic steatohepatitis (NASH) is a condition characterized by the buildup of fat in the liver, potentially leading to liver fibrosis, cirrhosis, liver failure or cancer, and has an especially high prevalence among people who are obese or have type 2 diabetes. NASH is estimated to affect approximately 1.5% to 6.5% of adults in the U.S.1
About RNAi and Dicerna’s GalXC RNAi Platform Technologies
Ribonucleic acid interference, or RNAi, provides a unique advantage to other disease inhibitor technologies, like small-molecule pharmaceuticals or monoclonal antibodies: instead of targeting proteins after they have been produced and released, RNAi silences the genes themselves via the targeted destruction of the messenger RNA (mRNA) made from the gene. Rather than seeking to inhibit a protein directly, the RNAi approach can prevent a disease-causing protein’s creation, directly impacting disease manifestation.
Dicerna’s proprietary GalXC RNAi platform aims to advance the development of next-generation RNAi-based therapies. Investigational therapeutics developed using our flagship GalXC technology utilize a proprietary N-acetyl-D-galactosamine (GalNAc)-mediated structure of double-stranded RNA molecules that are designed to bind specifically to receptors on liver cells, leading to selective hepatocyte internalization and access to the RNAi machinery within the cells. Dicerna is continuously innovating and exploring new applications for RNAi technology beyond GalNAc-mediated delivery to the liver, including alternative RNA structures and fully synthetic ligands that target other tissues and enable new therapeutic applications, referred to as GalXC-Plus.